Fabry Disease

New Studies Affirm Safety Profile, Efficacy of Agalsidase Beta in Fabry Disease

April 30th 2021, 12:50pm


About 1 in 3 patients experienced adverse drug reactions in the 2 new safety studies, aligning with earlier reports.

Analysis: Kidney Transplantation in Fabry Disease Has High Success Rate

April 28th 2021, 1:48pm


All-cause graft failure in kidney transplant recipients with Fabry disease was about 30%, and improved when enzyme replacement therapy became available.

Most Patients With Fabry Disease Face Mild or Moderate Symptoms Upon COVID-19 Infection

April 23rd 2021, 11:00am


Despite concerns that patients with Fabry disease might be at heightened risk of severe symptoms from SARS-CoV-2, early data find that most patients experience mild or moderate symptoms.

Overcoming Barriers to Genetic Testing Key to Identifying More Cases of Fabry Disease

April 21st 2021, 12:15pm


These national and regional barriers, which range from cost of screening to patient education, have hindered the widespread use of screening, say the researchers.

Omalizumab Enables Fabrazyme Desensitization After Anaphylaxis, Report Says

April 15th 2021, 1:46pm


This is a case study of a male patient with Fabry disease who presented with severe anaphylaxis following re-initiation of full-strength treatment with intravenous agalsidase beta (Fabrazyme).

PAN Foundation Announces New Fabry Disease Assistance Fund

January 26th 2019, 3:00am


The Patient Access Network (PAN) Foundation has opened a new patient assistance program for people living with Fabry disease/

Gene Therapy for Fabry Disease to Advance to Phase 2 Clinical Trials

February 4th 2018, 6:00pm


Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).